Revlimid® (lenalidomide) is an FDA-approved small molecule drug developed and marketed by Celgene for the treatment of multiple myeloma (MM) in combination with dexamethasone and for the treatment of myelodysplastic syndromes in patients with a specific 5q chromosomal deletion. The cellular target and mechanism of action of Revlimid® are unknown in the art. In 2009, sales of Revlimid® reached USD $ 1.7 billion and sales in 2010 are expected to exceed USD $2.0 billion, making Revlimid® one of the most successful oncology products introduced into commerce in the past five years. Based on this demonstrated commercial success and the absence of any FDA-approved competitive therapeutic drugs with a similar mechanism of action, the potential economic value of the demonstrated anti-cancer target(s) of Revlimid® (lenalidomide) is considerable.
Revlimid® belongs to a class of compounds referred to as immunomodulatory drugs (IMiDs) that include the analogs thalidomide (FDA-approved for MM and leprosy) and pomalidomide (Actimid™, under development by Celgene). Thomson Pharma has projected that annual Revlimid® drug sales could reach USD $3.8 billion by 2013. Because of thalidomide's well-known teratogenic effects, Revlimid® is sold under an FDA-mandated risk mitigation program with a ‘Black Box Warning’ describing the risks of birth defects. Recently, the protein target responsible for the teratogenicity of thalidomide was published as Cereblon (CRBN) (Ito et al, Science 327:1345-1350 (2010)); however, the anticancer target(s) of IMiD's are still unknown.